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Scientists have developed a provocative new technique for correcting tiny but potentially devastating errors in mutant genes, an approach they hope will someday provide a strategy for treating at least some genetic disorders.

The approach, known as chimeraplasty or targeted gene correction, is still in its infancy and faces many technical challenges before it is clear whether the method can move from bench to bedside. Moreover, some skeptics say that a number of investigators have had difficulty getting the procedure to work and question whether the approach will be generally applicable to a variety of cell types.

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Cells from albino mice have a genetic mutation that prevents them from making an enzyme (tyrosinase) necessary to produce the black pigment melanin (left), but descendants of an albino cell with a tyrosinase gene successfully repaired by chimeraplasty are readily able to make melanin (right). Photo credit: Vitali Alexeev, PhD, and Kyonggeun Yoon, PhD

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